I am sure many of the members of the society are aware that the research team at Prince Henry/Prince of Wales Hospitals has conducted a number of "randomised, double blind, placebo-controlled trials" assessing potential treatment options for patients with CFS. I would like to discuss with you therefore what such a trial means both for the patients and for the researchers.

Firstly, what do the words mean?: A "randomised" study is one in which usually two possible drugs are given to the participants - one is the active drug which is believed to hold some promise as an effective treatment, the other is a "placebo" - a drug which has no expected treatment value and which looks, tastes, feels as close as possible to identical to the active drug.

The randomisation is a code (usually generated by computer) which allocates each individual by chance to receive either the active drug or the placebo. This aspect of the trial designed tends to make up for any variation in the nature of the illness affecting the patients enrolled (such as differences in severity or duration of symptoms), and therefore allows successful statistical evaluation of any difference response rate between the two groups.

The "double-blind" is the procedure in the study design which makes every attempt to ensure that neither the patient nor the researchers are aware which drug (active or placebo) each subject is receiving.

The "blind" is very important in this type of research as it ensures that neither the patients nor the researcher can have any direct effect on the outcome of the treatment due to factors such as the effect of believing that one is receiving the active drug which in itself may produce some benefit.

Similarly, the researchers may inadvertently or otherwise affect the outcome it he is aware of which treatment a patient is receiving.

Every patient is monitored in an identical fashion (in our trials, with report forms of symptoms and ability to participate in daily activities as well as immunological testing).

At the completion of the trial, that is when every patient has completed the treatment course and completed the follow-up, a code is broken which details which patients have received the active and which patients the placebo treatment. An analysis is then made of the data which has already been collected for each patient, as to whether a larger number of patients receiving the active drug than the placebo showed a response to the treatment.

Patients who enrol at the beginning of a large trial may have to wait a long period until all the following patients have completed the trial and the code is broken to find out which treatment they have been given. It is usual practise to offer the active drug to all placebo recipients after the trial if the statistical analysis of the data shows the drug is effective.

This type if trial design is the only accepted way of establishing a new treatment into medical practice. It is highly demanding for both patients and researchers, and carries with it inevitable difficulties such as the stress of the uncertainty of which treatment has been received.

Drugs currently used in the treatment of patients with CFS, which have not been submitted to this rigorous scientific scrutiny (such as vitamin C, or anti-candida agents, homeopathy etc) are unlikely to ever be accepted in conservative medical practice (although they may or may not have any true treatment value).

After the completion of a trial and the analysis of the data, there is an inevitable delay of 12-24 months before the publication of results occurs in the medical literature. During this time the researchers cannot provide details of the outcome of the trial to the patients or the public, as this would represent a breach of copyright of any subsequent publication.

It is only with the publication of the results of a trial (and often the completion of a second trial confirming the results of the first) that use of the drug evaluated is likely to become available or accepted for usage in medical practice.

Reprinted from M.E. and You, Newsletter of the M.E./C.F.S. Soc. of N.S.W. Inc. Written by Dr. Andrew Lloyd.

Reprinted from Emerge, June 1990.